Olry de Labry-Lima Antonio, Ponce-Polo Angela, García-Mochón Leticia, Ortega-Ortega Marta, Pérez-Troncoso Daniel, Epstein David
Escuela Andaluza de Salud Pública, Granada, Spain; Instituto de Investigación Biosanitaria Ibs, Granada, Spain; CIBER en Epidemiología and Salud Pública (CIBERESP), Spain.
Andalusian Network for the Design & Translation of Advanced Therapies, Sevilla, Spain.
Value Health. 2023 Jan;26(1):138-150. doi: 10.1016/j.jval.2022.07.004. Epub 2022 Aug 26.
Advanced therapy medicinal products (ATMPs) are drugs for human use for the treatment of chronic, degenerative, or life-threatening diseases that are based on genes, tissues, or cells. This article aimed to identify and critically review published economic analyses of ATMPs.
A systematic review of economic analyses of ATMPs was undertaken. Study characteristics, design, sources of data, resources and unit costs, modeling and extrapolation methods, study results, and sensitivity analyses were assessed.
A total of 46 economic analyses of ATMP (from 45 articles) were included; 4 were cell therapy medicinal products, 33 gene therapy medicinal products, and 9 tissue-engineered products. 30 therapies had commercial marketing approval; 39 studies were cost-utility analysis, 5 were cost-effectiveness analysis, and 2 were cost only studies. Four studies predicted that the ATMP offered a step change in the management of the condition and 10 studies estimated that the ATMP would offer a lower mean cost.
Comparison with historical controls, pooling of data, and use of techniques such as mixture cure fraction models should be used cautiously. Sensitivity analyses should be used across a plausible range of prices. Clinical studies need to be designed to align with health technology assessment requirements, including generic quality of life, and payers should aim for clarity of criteria. Regulators and national payers should aim for compatibility of registers to allow interchange of data. Given the increasing reliance on industry-funded economic analyses, careful critical review is recommended.
先进治疗药品（ATMPs）是用于治疗慢性、退行性或危及生命疾病的人用药物，其基于基因、组织或细胞。本文旨在识别并严格审查已发表的ATMPs经济分析。
对ATMPs的经济分析进行了系统综述。评估了研究特征、设计、数据来源、资源和单位成本、建模和外推方法、研究结果以及敏感性分析。
共纳入46项ATMPs的经济分析（来自45篇文章）；4项为细胞治疗药品，33项为基因治疗药品，9项为组织工程产品。30种疗法获得了商业销售批准；39项研究为成本效用分析，5项为成本效果分析，2项仅为成本研究。4项研究预测ATMP在疾病管理方面带来了重大变革，10项研究估计ATMP将提供更低的平均成本。
与历史对照的比较、数据汇总以及混合治愈分数模型等技术的使用应谨慎。应在合理的价格范围内进行敏感性分析。临床研究的设计应符合卫生技术评估要求，包括通用生活质量，支付方应明确标准。监管机构和国家支付方应致力于登记册的兼容性，以实现数据的互换。鉴于对行业资助的经济分析的依赖日益增加，建议进行仔细的严格审查。
